The ALS Therapy Development Institute, medical charity LifeArc and life science company Axol Bioscience have launched patient induced pluripotent stem cell (iPSC)-based research to improve sporadic ALS modelling (PRISM ALS).
“Our hope is that the stem cell models we produce can unleash a new generation of treatments that could be effective against this disease by slowing its progression and, ultimately, curing it,” said LifeArc head of MND Paul Wright. “We need to do more for people living with MND/ALS, and PRISM ALS brings together leading organisations to help make that happen.”
Current treatments do not reflect the diverse nature of ALS. Although nearly 85% of ALS cases are sporadic, much of ALS drug discovery relies on models representing a limited number of rare genetic subtypes. This constrains target discovery, limits therapeutic testing across patient populations and contributes to the high failure rate of clinical trials.
The initiative aims to expand access to high-quality, patient-derived stem cell models that better reflect the biological complexity of the disease. According to PRISM ALS, the effort will provide a reliable, high-quality and accessible source of sporadic ALS/MND models for research to develop and evaluate a diverse panel of well-characterised, patient-derived iPSC models capturing both genetic and sporadic forms of ALS.
“By characterising iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery,” said ALS TDI CEO and chief scientific officer Dr Fernando Vieira.
The stem cells used in PRISM ALS are derived from samples contributed by people living with ALS through ALS TDI’s ALS Research Collaborative (ARC) Study, which is the longest-running longitudinal patient study in ALS.
Such standardised, human-relevant models could allow researchers and drug developers to better understand disease mechanisms, identify therapeutic targets and evaluate treatments across distinct biological subtypes. Therapies could be tested in models that mirror their own biology more closely, increasing the likelihood that discoveries will translate into effective treatment.
By enabling standardised production at scale, the collaboration supports quality, consistency, and reproducibility across laboratories.
“We’re delighted to participate in this consortium to develop multiple iPSC-derived end point cell types from sporadic ALS iPSC lines that reflect for the first time, real-world variability across age, sex, and genotype,” said Axol Bioscience head of scientific programmes Sapna Vyas. “By leveraging Axol’s scalable manufacturing infrastructure, we will facilitate access to standardised iPSC-derived cells that empower researchers to stratify patients, assess subgroup responses to therapies, and reduce late-stage clinical trial failures.”
